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Current Strategies of Muscular Dystrophy Therapeutics: An Overview
Muscular dystrophies are a group of geneticGenetics disorders characterized by varying degrees of progressive muscle weakness and degeneration. They...
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Correction to: Direct Reprogramming of Adult Human Cardiac Fibroblasts into Induced Cardiomyocytes Using miRcombo
This chapter was previously published as non-Open Access. Under the request of the authors the chapter is now converted to Open Access and is...
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Develo** Therapeutic Splice-Correcting Antisense Oligomers for Adult-Onset Pompe Disease with c.-32-13T>G Mutation
The mutation c.-32-13T>G in the GAA gene impacts normal exon 2 splicing and is found in two-thirds of late-onset Pompe disease cases. We have...
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CRISPR-Cas9 Correction of Duchenne Muscular Dystrophy in Mice by a Self-Complementary AAV Delivery System
Duchenne muscular dystrophy (DMD) is a fatal neuromuscular disorder, caused by mutations in the DMD gene coding dystrophin. Applying clustered...
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Cardiac and Skeletal Muscle Pathology in the D2/mdx Mouse Model and Caveats Associated with the Quantification of Utrophin
Duchenne muscular dystrophy (DMD)Duchenne muscular dystrophy (DMD) (the most common form of muscular dystrophyMuscular dystrophies) is caused by a...
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Restoring Dystrophin Expression with Exon 44 and 53 Skip** in the DMD Gene in Immortalized Myotubes
Phosphorodiamidate morpholino oligomer (PMO)-mediated exon skip** is a therapeutic approach that applies to many Duchenne muscular dystrophy (DMD)...
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Use of Glycine to Augment Exon Skip** and Cell Therapies for Duchenne Muscular Dystrophy
Antisense oligonucleotide (AO)-based exon-skip** and cell therapies are the main therapeutic approaches for Duchenne muscular dystrophy (DMD)....
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High-Throughput Screening to Identify Modulators of Sarcospan
High-throughput screening enables the discovery of disease-modifying small molecules. Here, we describe the development of a scalable, cell-based...
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Quantification of Muscle Satellite Stem Cell Divisions by High-Content Analysis
High-content screeningScreening is commonly performed on 2D cultured cells, which is high throughputHigh throughput but has low biological relevance....
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Identifying FDA-Approved Drugs that Upregulate Utrophin A as a Therapeutic Strategy for Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a neuromuscular disease caused by mutations and deletions within the DMD gene, which result in a lack of...
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Restoring Dystrophin Expression by Skip** Exons 6 and 8 in Neonatal Dystrophic Dogs
Duchenne muscular dystrophy (DMD) is caused by the mutations in the DMD gene resulting in no dystrophin production. Skip** DMD exons using...
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Assessment of the Gene Therapy Immune Response in the Canine Muscular Dystrophy Model
The immune response is a primary hurdle in the development of gene therapy for neuromuscular diseases. Both innate and adaptive immune responses have...
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Molecular and Biochemical Assessment of Gene Therapy in the Canine Model of Duchenne Muscular Dystrophy
Mutations in the dystrophin gene result in Duchenne muscular dystrophy (DMD), a progressive muscle-wasting disease. Adeno-associated virus (AAV)...
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Preparation of NanoMEDIC Extracellular Vesicles to Deliver CRISPR-Cas9 Ribonucleoproteins for Genomic Exon Skip**
The CRISPR-Cas9 system has quickly become the standard tool for genome editing. To deliver this system to target cells, adeno-associated virus (AAV)...
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Knocking Down DUX4 in Immortalized Facioscapulohumeral Muscular Dystrophy Patient-Derived Muscle Cells
The third most common muscular dystrophy in the world, facioscapulohumeral muscular dystrophy (FSHD), is an inherited disorder characterized by...
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Rapid Freezing of Skeletal and Cardiac Muscles Using Isopentane Cooled with Liquid Nitrogen and Tragacanth Gum for Histological, Genetic, and Protein Expression Studies
Histological and molecular genetic evaluation of skeletal and cardiac muscles is an indispensable part of understanding muscle biology and the...
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Monitoring Plasma Membrane Injury-Triggered Endocytosis at Single-Cell and Single-Vesicle Resolution
Plasma membrane injury activates membrane trafficking and remodeling events that are required for the injured membrane to repair. With the rapidity...
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